Advertisement
| cystic fibrosis enzyme replacement therapy: Textbook of Pediatric Gastroenterology, Hepatology and Nutrition Stefano Guandalini, Anil Dhawan, David Branski, 2015-09-30 This textbook provides a comprehensive and state-of-the-art overview of the major issues specific to the field of pediatric gastroenterology, hepatology, and nutrition. The first part of the book, Gastroenterology and Nutrition, presents in a systematic way the overall scope of issues encountered by children (newborn to teenagers) suffering from disorders of the gastrointestinal tract, pancreas and/or presenting nutritional issues. These chapters are structured in logical sections to facilitate consultation and include major topics ranging from congenital disorders to gastrointestinal problems of the newborn, infectious diseases of the gastrointestinal tract, and approach to nutritional problems in the various pediatric ages. The second part of the book, Hepatology, is articulated in a series of chapters which present a comprehensive review of congenital and acquired disorders of the biliary tract and liver. This section also includes a critical analysis of available diagnostic and therapeutic procedures and future perspectives. Written by experts in the field, Textbook of Pediatric Gastroenterology, Hepatology and Nutrition: A Comprehensive Guide to Practice constitutes a much needed, innovative resource combining updated, reliable and comprehensive information with agile consultation for a streamlined approach to the care of children with such disorders. |
| cystic fibrosis enzyme replacement therapy: Clinical Pancreatology for Practising Gastroenterologists and Surgeons Juan Enrique Dominguez-Munoz, 2021-03-17 Clinical Pancreatology Since the book Clinical Pancreatology for Practising Gastroenterologists and Surgeons was first published sixteen years ago, the knowledge and clinical management of pancreatic diseases have developed markedly. Thanks to the development of the translational research and the from bench to bedside concept, much progress from the lab has been applied to clinical practice. In addition, several highly relevant clinical trials published over the last years have resulted in the update and optimisation of clinical guidelines. A new and validated classification of severity and complications of acute pancreatitis is firmly rooted in clinical practice and has been the basis for the development of minimally invasive approaches to pancreatic necrosis. The etiopathogenic knowledge of chronic pancreatitis and other pancreatopaties, like that associated with diabetes mellitus, has developed significantly. Especially important has been the development of the field of cystic pancreatic tumours, which has been reflected in the publication of several guidelines and consensus reports over the last few years. Most research efforts have focused on pancreatic cancer, which have led and will further lead to a significant increase in the therapeutic armamentarium against this devastating disease. Finally, many newly published studies have changed the concept, causes, clinical relevance, diagnosis and treatment of exocrine pancreatic insufficiency. This new edition of Clinical Pancreatology for Practising Gastroenterologists and Surgeons has enjoyed the collaboration of the world’s leading experts in each of the areas of clinical pancreatology with the aim of facilitating gastroenterologists, surgeons, oncologists, internists, nutritionists, diabetologists, paediatricians, radiologists, pathologists and other specialists in their decision making when facing patients with pancreatic diseases in their daily clinical practice. All in all, this book supplies an indispensable update of the relevant aspects of clinical pancreatology. |
| cystic fibrosis enzyme replacement therapy: Landmark Papers in Internal Medicine Harold C. Sox, Edward J. Huth, American College of Physicians (2003- ), 2009 ACPs journal, Annals of Internal Medicine, is one of the most prestigious journals in medicine. This new book looks at the landmark papers published in Annals, as selected by leading experts from each subspecialty of internal medicine, and how they impacted (and continue to influence) medical science. |
| cystic fibrosis enzyme replacement therapy: Emerging Therapeutic Approaches for Cystic Fibrosis Miquéias Lopes-Pacheco, Nicoletta Pedemonte, Anthony Kicic, 2020-01-27 |
| cystic fibrosis enzyme replacement therapy: Liver Disease in Children Frederick J. Suchy, Ronald J. Sokol, William F. Balistreri, 2007-05-07 Completely revised new edition of the premier reference on pediatric liver disease. Liver Disease in Children, 3rd Edition provides authoritative coverage of every aspect of liver disease affecting infants, children, and adolescents. The book offers an integrated approach to the science and clinical practice of pediatric hepatology and charts the substantial progress in understanding and treating these diseases. Chapters are written by international experts and address the unique pathophysiology, manifestations, and management of these disorders in the pediatric population. The third edition has been thoroughly updated and features new contributions on liver development, cholestatic and autoimmune disorders, fatty liver disease, and inborn errors of metabolism. With the continued evolution of pediatric hepatology as a discipline, this text remains an essential reference for all physicians involved in the care of children with liver disease. |
| cystic fibrosis enzyme replacement therapy: Pediatric Nutrition in Practice B. Koletzko, J. Bhatia, Z.A. Bhutta, P. Cooper, M. Makrides, R. Uauy, W. Wang, 2015-04-17 There is no other time in life when the provision of adequate and balanced nutrition is of greater importance than during infancy and childhood. During this dynamic phase characterized by rapid growth, development and developmental plasticity, a sufficient amount and appropriate composition of nutrients both in health and disease are of key importance for growth, functional outcomes such as cognition and immune response, and the metabolic programming of long-term health and well-being. This compact reference text provides concise information to readers who seek quick guidance on practical issues in the nutrition of infants, children and adolescents. After the success of the first edition, which sold more than 50'000 copies in several languages, the editors prepared this thoroughly revised and updated second edition which focuses again on nutritional challenges in both affluent and poor populations around the world. Serving as a practical reference guide, this book will contribute to further improving the quality of feeding of healthy infants and children, as well as enhancing the standards of nutritional care in sick children. |
| cystic fibrosis enzyme replacement therapy: Foundations of Respiratory Medicine Simon Hart, Mike Greenstone, 2018-09-04 This book meets the need for a resource that covers the core knowledge required to pass the SCE exam, which includes the broad field of respiratory medicine. This book is also highly applicable to core medical trainees sitting their MRCP examinations. The format is ideal for effective exam revision with individual chapters covering the key points of each condition in sufficient (but not excessive) detail. Examples of imaging (CXR, CT, PET-CT) are utilised to illustrate cases and descriptions of modern respiratory intervention such as the EBUS/EUS-guided sampling and medical thoracoscopy is included in this essential exam resource. |
| cystic fibrosis enzyme replacement therapy: Therapeutic Enzymes: Function and Clinical Implications Nikolaos Labrou, 2019-09-03 Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy. This volume aims to overview these therapeutic enzymes, focusing in particular on more recently approved enzymes produced by recombinant DNA technology. This volume is composed of four sections. Section 1 provides an overview of the production process and biochemical characterization of therapeutic enzymes, while Section 2 focuses upon the engineering strategies and delivery methods of therapeutic enzymes. Section 3 highlights the clinical applications of approved therapeutic enzymes, including aspects on their structure, indications and mechanisms of action. Together with information on these mechanisms, safety and immunogenicity issues and various adverse events of the recombinant enzymes used for therapy are discussed. Section 4, provides discussion on the prospective and future developments of new therapeutic enzymes. This book is aimed at academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules. |
| cystic fibrosis enzyme replacement therapy: Nutrition in Cystic Fibrosis Elizabeth H. Yen, Amanda Radmer Leonard, 2015-11-28 Nutritional therapies have been key early interventions, and remain central to the well-being and survival of patients with cystic fibrosis. The nature of the disease causes significant alterations in a patient’s ability to process and assimilate nutrients. Furthermore, many factors contribute to higher metabolic demands throughout a patient’s life. In combination, maldigestion, malabsorption, and increased metabolic demands pose a high hurdle for the patient to overcome in order to maintain optimal nutritional status. Nutrition in Cystic Fibrosis: A Guide for Clinicians is an excellent resource for physicians, nurses and dietitians who deliver care for patients with cystic fibrosis. The book provides an introduction to cystic fibrosis and nutritional assessments. It will also serve as a comprehensive guide to the nutritional monitoring and management of patients with cystic fibrosis including special populations within cystic fibrosis that require additional considerations. The chapters are written by experts in their fields and include the most up to date scientific and clinical information. Nutrition in Cystic Fibrosis: A Guide for Clinicians targets pediatric and adult pulmonologists and gastroenterologists, residents and fellows, internists, pediatricians, nurses, dietitians and general practitioners who treat patients with cystic fibrosis. |
| cystic fibrosis enzyme replacement therapy: Alex Frank Deford, 2015-02-24 A father’s moving memoir of cystic fibrosis “captures a brave child’s legacy as well as the continuing fight against the genetic disease” (The New York Times). In 1971 a girl named Alex was born with cystic fibrosis, a degenerative genetic lung disease. Although health-care innovations have improved the life span of CF patients tremendously over the last four decades, the illness remains fatal. Given only two years to live by her doctors, the imaginative, excitable, and curious little girl battled through painful and frustrating physical-therapy sessions twice daily, as well as regular hospitalizations, bringing joy to the lives of everyone she touched. Despite her setbacks, brave Alex was determined to live life like a typical girl—going to school, playing with her friends, traveling with her family. Ultimately, however, she succumbed to the disease in 1980 at the age of eight. Award-winning author Frank Deford, celebrated primarily as a sportswriter, was also a budding novelist and biographer at the time of his daughter’s birth. Deford kept a journal of Alex’s courageous stand against the disease, documenting his family’s struggle to cope with and celebrate the daily fight she faced. This book is the result of that journal. Alex relives the events of those eight years: moments as heartwarming as when Alex recorded herself saying “I love you” so her brother could listen to her whenever he wanted, and as heartrending as the young girl’s tragic, dawning realization of her own very tenuous mortality, and her parents’ difficulty in trying to explain why. Though Alex is a sad story, it is also one of hope; her greatest wish was that someday a cure would be found. Deford has written a phenomenal memoir about an extraordinary little girl. |
| cystic fibrosis enzyme replacement therapy: Medical Management of the Surgical Patient E-Book Geno J. Merli, Howard H. Weitz, 2008-04-08 This practical handbook delivers complete, to-the-point, evidence-based guidance on the preoperative, perioperative, and post-operative medical care of surgical patients. Each chapter focuses on a particular area of clinical concern, with concise presentations of pathophysiology, assessment and management options, the latest drug treatment information, and essential information on risk stratification and quality improvement. The result is an invaluable source on the management of surgical patients with co-existing medical problems that may be affected by surgery, as well as how to approach medical complications that may occur during or following surgical procedures. Comprehensive discussions at the beginning of each chapter emphasize consultation in surgical patient management. A concise, bulleted format lets you absorb key information at a glance. Extensively updated chapters. Easy-to-read tables present key information on each disorder, including classification, causes, risk factors and indexes, drug treatment information, mortality rates, laboratory findings, and postoperative complications. Recommendations accompanied by an assessment of the quality of supportive evidence, including randomized controlled trials, population based reviews, and consensus guidelines. A topical index takes you immediately to the answers you need. A new, smaller design makes the book easy to carry with you anywhere. |
| cystic fibrosis enzyme replacement therapy: Metabolic Diseases E. Gilbert-Barness, L.A. Barness, P.M. Farrell, 2017-01-06 The 2nd Edition of Metabolic Diseases provides readers with a completely updated description of the Foundations of Clinical Management, Genetics, and Pathology. A distinguished group of 31 expert authors has contributed 25 chapters as a tribute to Enid Gilbert-Barness and the late Lewis Barness--- both pioneers in this topic. Enid’s unique perspectives on the pathology of genetic disorders and Lew’s unsurpassed knowledge of metabolism integrated with nutrition have inspired the contributors to write interdisciplinary descriptions of generally rare, and always challenging, hereditary metabolic disorders. Discussions of these interesting genetic disorders are organized in the perspective of molecular abnormalities leading to morphologic disturbances with distinct pathology and clinical manifestations. The book emphasizes recent advances such as development of improved diagnostic methods and discovery of new, more effective therapies for many of the diseases. It includes optimal strategies for diagnosis and information on access to specialized laboratories for specific testing. The target audience is a wide variety of clinicians, including pediatricians, neonatologists, obstetricians, maternal-fetal specialists, internists, pathologists, geneticists, and laboratorians engaged in prenatal and/or neonatal screening. In addition, all scientists and health science professionals interested in metabolic diseases will find the comprehensive, integrated chapters informative on the latest discoveries. It is our hope that the 2nd Edition will open new avenues and vistas for our readers and that they will share with us the interest, excitement and passion of the research into all these challenging disorders. |
| cystic fibrosis enzyme replacement therapy: Newborn Screening for Pompe Disease Wuh-Liang Hwu, Yin-Hsiu Chien, Raymond Wang, 2021-09-02 Pompe disease, also known as acid maltase deficiency or acid alpha-glucosidase deficiency, in its most severe form results in a rapidly progressive, neonatal-onset skeletal and cardiomyopathy, leading to early infantile death without treatment. The development of treatment with recombinant enzyme replacement therapy radically transformed the clinical trajectory of those affected, enabling long-term ventilator-free survival with resolution of cardiomyopathy. These positive clinical outcomes resulted in the implementation of newborn screening programs for Pompe disease across the world. This Special Issue highlights some of the experiences of Pompe screening programs worldwide and discusses public policy and ethical issues elicited by presymptomatic screening for Pompe disease. |
| cystic fibrosis enzyme replacement therapy: Clinical Rounds in Hepatology Virendra Singh, Akash Roy, 2022-01-29 This book presents a comprehensive collection of classic cases and problem scenarios encountered as bedside case discussions during ward rounds. It facilitates the practical management of hepatic disorders. The unique aspect of hepatology involves the blend of the basic tenet of clinical medicine applied to liver disease and a multitude of interventional modalities in the management of liver, biliary, and pancreatic diseases. This book takes the reader through the process of ruling in and ruling out possibilities based on clinical data (history and examination) and then traces the logical trajectory of each case from recommended investigations to the analysis of test results and finally to making a syndromic diagnosis. By adopting an evidence-based approach, the book emphasizes analytical and need-based studies to exclude any mimics. This book helps practice hepatologists and gastroenterologists for a systematic approach towards the most common cases. |
| cystic fibrosis enzyme replacement therapy: An Illustrated Guide to Pediatric Surgery Ahmed H. Al-Salem, 2014-09-24 Written in a simple point by point style for ease of use, this volume covers all aspects of pediatric surgery with emphasis on important points for diagnosis and management. Each chapter covers a topic with emphasis on the most common conditions in neonatal and general pediatric surgery. The text is well illustrated with clinical, operative, radiological, and histopathological color figures and illustrations. The book also presents some of the rare conditions encountered in pediatric surgery, as well as common pediatric urology conditions. An Illustrated Guide to Pediatric Surgery is a useful reference to pediatric surgeons, specialists, fellows and residents, as well as general surgeons, pediatricians, neonatologists, medical students and interns interested in pediatric surgery. |
| cystic fibrosis enzyme replacement therapy: Immunopharmacology Manzoor M. Khan, 2008-12-19 During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology. |
| cystic fibrosis enzyme replacement therapy: A Life Course Perspective on Health Trajectories and Transitions Claudine Burton-Jeangros, Stéphane Cullati, Amanda Sacker, David Blane, 2015-08-11 This open access book examines health trajectories and health transitions at different stages of the life course, including childhood, adulthood and later life. It provides findings that assess the role of biological and social transitions on health status over time. The essays examine a wide range of health issues, including the consequences of military service on body mass index, childhood obesity and cardiovascular health, socio-economic inequalities in preventive health care use, depression and anxiety during the child rearing period, health trajectories and transitions in people with cystic fibrosis and oral health over the life course. The book addresses theoretical, empirical and methodological issues as well as examines different national contexts, which help to identify factors of vulnerability and potential resources that support resilience available for specific groups and/or populations. Health reflects the ability of individuals to adapt to their social environment. This book analyzes health as a dynamic experience. It examines how different aspects of individual health unfold over time as a result of aging but also in relation to changing socioeconomic conditions. It also offers readers potential insights into public policies that affect the health status of a population. |
| cystic fibrosis enzyme replacement therapy: Handbook of Drug Administration via Enteral Feeding Tubes, 3rd edition Rebecca White, Vicky Bradnam, 2015-03-11 With over 400 drug monographs, this book covers the technical, practical and legal aspects that you should consider before prescribing or administering drugs via enteral feeding tubes. |
| cystic fibrosis enzyme replacement therapy: Pediatric Thoracic Surgery D.H. Parikh, David Crabbe, Alex Auldist, Steven Rothenberg, 2009-03-26 This text covers new innovations and concepts in pediatric thoracic surgery practice, basic science and evidence, and the technical aspects of common and rare operative procedures. It is essential for pediatric surgical trainees and consultants with interest in pediatric thoracic surgery. Providing comprehensive coverage of newer developments, it is also a useful reference work for pediatric and thoracic surgeons and a valuable guide for surgeons (adult or pediatric) managing pediatric thoracic surgery on occasional basis or only during acute emergency.Covering the subjects within pediatric thoracic surgery (non-cardiac) in significant depth, this book acts as a reference text for consultants undertaking pediatric thoracic work as well as for pediatric respiratory, anesthetists and fetal medicine doctors. Topics within this book will also be of interest to pediatric respiratory physicians and pediatric oncologists. |
| cystic fibrosis enzyme replacement therapy: Pancreatic Disease Paul G. Lankisch, Eugene P. DiMagno, 1999-03-29 This symposium volume summarizes the latest research, research that has greatly advanced our understanding of both the basic mechanisms of pancreatic physiology and pathophysiology and the diagnosis and treatment of the diseases of the exocrine pancreas. By doing so, it serves to give direction to future research. Diseases discussed include acute and chronic pancreatitis, cystic fibrosis, and pancreatic cancer. The volume also reflects a central concern of the symposium: to stimulate young persons embarking on a career in this area. |
| cystic fibrosis enzyme replacement therapy: Therapeutic, Probiotic, and Unconventional Foods Alexandru Mihai Grumezescu, Alina Maria Holban, 2018-04-18 Therapeutic, Probiotic and Unconventional Foods compiles the most recent, interesting and innovative research on unconventional and therapeutic foods, highlighting their role in improving health and life quality, their implications on safety, and their industrial and economic impact. The book focuses on probiotic foods, addressing the benefits and challenges associated with probiotic and prebiotic use. It then explores the most recently investigated and well-recognized nutraceutical and medicinal foods and the food products and ingredients that have both an impact on human health and a potential therapeutic effect. The third and final section explores unconventional foods and discusses intriguing and debated foods and food sources. While research has been conducted on the beneficial biological effects of probiotics and therapeutic food, the use of these foods remains controversial. To overcome the suspicion of the use of alternative, homeopathic and traditional products as therapy, this book reveals and discusses the most recent and scientifically sound and confirmed aspects of the research. - Compiles the most recent, interesting and innovative research on unconventional and therapeutic foods - Highlights the role of unconventional and therapeutic foods in improving health and life quality - Discusses the implications of unconventional and therapeutic foods on safety - Presents the industrial and economic impact of unconventional and therapeutic foods |
| cystic fibrosis enzyme replacement therapy: Fabry Disease Deborah Elstein, Gheona Altarescu, Michael Beck, 2010-08-02 Fabry disease is an X-linked inborn error of metabolism wherein deficiency of a lysosomal enzyme results in systemic deposition of glycosphingolipids. Storage deposition, and hence pathological disease, occurs preferentially in renal glomerular and tubular epithelial cells, myocardial cells, heart valve fibrocytes, neurons of dorsal root ganglia, and in endothelial smooth muscle cells of blood vessels. Thus, Fabry disease is a multi-system disorder, albeit with considerable phenotypic heterogeneity in onset and in severity; however, it is progressive, exhibits extensive morbidity, and is life-threatening. Within the past two decades, there has been a radical change in the natural course Fabry disease by virtue of the availability of specific enzyme replacement therapy. Moreover, there has been a concerted effort to better understand the underlying pathology and equally to identify patients prior to the onset of irreversible end-organ damage. It is to be hoped that the future for patients with Fabry disease can be viewed with greater, albeit guarded, optimism. This state-of-the-art textbook attempts to bridge the span of pre-clinical studies, clinical finding, and management options in a readable but comprehensive manner for the medical practitioner as well as the interested non-medical reader. |
| cystic fibrosis enzyme replacement therapy: FDA's Drug Review Process and the Package Label Tom Brody, 2017-12-01 FDA's Drug Review Process and the Package Label provides guidance to pharmaceutical companies for writing FDA-submissions, such as the NDA, BLA, Clinical Study Reports, and Investigator's Brochures. The book provides guidance to medical writers for drafting FDA-submissions in a way more likely to persuade FDA reviewers to grant approval of the drug. In detail, the book reproduces data on efficacy and safety from one hundred different FDA-submissions (NDAs, BLAs). The book reproduces comments and complaints from FDA reviewers regarding data that are fragmentary, ambiguous, or that detract from the drug's approvability, and the book reveals how sponsors overcame FDA's concerns and how sponsors succeeded in persuading FDA to grant approval of the drug. The book uses the most reliable and comprehensive source of information available for writing FDA-submissions, namely text and data from NDAs and BLAs, as published on FDA's website. The source material for writing this book included about 80,000 pages from FDA's Medical Reviews, FDA's Clinical Pharmacology Reviews, and FDA's Pharmacology Reviews, from one hundred different NDAs or BLAs for one hundred different drugs. Each chapter focuses on a different section of the package label, e.g., the Dosage and Administration section or the Drug Interactions section, and demonstrates how the sponsor's data supported that section of the package label. - Reveals strategies for winning FDA approval and for drafting the package label - Examples are from one hundred FDA-submissions (NDAs, BLAs) for one hundred different drugs, e.g., for oncology, metabolic diseases, autoimmune diseases, and neurological diseases - This book uses the most reliable and comprehensive source of information available for writing FDA-submissions, namely, the data from NDAs and BLAs as published on FDA's website at the time FDA grants approval to the drug |
| cystic fibrosis enzyme replacement therapy: Hematopoietic Stem Cell Transplantation in Clinical Practice Jennifer G. Treleaven, A. John Barrett, 2008-09-02 A guide to the practice of stem cell transplantation, its status in the treatment of various disorders and the problems that arise after transplantation, aimed at the whole transplant team. - An up to date guide to best practice in the use of stem cell transplantation, covering current status in the treatment of malignant and non-malignant conditions, practical aspects and problems such as infection and graft versus host disease. - Has a practical, accessible approach with free use of algorithms, list tables. - Aimed at the whole transplant team - this is an interdisciplinary field. - International contributor team with editors in the UK and USA. - Illustrated in colour throughout. |
| cystic fibrosis enzyme replacement therapy: Enzymes as Drugs John S. Holcenberg, Joseph Roberts, 1981 |
| cystic fibrosis enzyme replacement therapy: Pediatric Respiratory Medicine Lynn Max Taussig, Louis I. Landau, 2008-01-01 This user-friendly text presents current scientific information, diagnostic approaches, and management strategies for the care of children with acute and chronic respiratory diseases. A consistent chapter format enables rapid and effortless location of the most current protocols on manifestations, etiologies, triggers, approaches to treatment, complications, and preventative strategies. Includes guidance on differential diagnosis to help determine which disease or condition the patient may have. Uses extensive color-coded algorithms to facilitate quick diagnosis, management, and treatment decisions. Provides the latest scientific information and diagnostic and management strategies for the care of children with respiratory illnesses. Presents cutting-edge coverage with new information on the biology of, and the influences on, the respiratory system during childhood, as well as the diagnosis and management of both common (ie, wheezing infant, cystic fibrosis, tuberculosis) and. |
| cystic fibrosis enzyme replacement therapy: Pediatric Respiratory Diseases Pablo Bertrand, Ignacio Sánchez, 2020-01-31 This is a comprehensive and authoritative textbook on pediatric pulmonology. Edited by Pablo Bertrand and Ignacio Sánchez, renowned academics and pediatricians from the Pontifical Catholic University of Chile, it encompasses five sections and 74 chapters, presenting and discussing the most important topics related to pediatric respiratory diseases. Written and presented in a simple and didactic format, it intends to ease learning and settlement of doubts in pediatric respiratory diseases. The reader is naturally introduced into the physiology, diagnosis, syndromes, diseases and the treatment associated with the respiratory pathologies affecting children. The chapters include algorithms for the treatment of various syndromes and updated treatment proposals grounded in evidence-based medicine for more than 50 pulmonary diseases. Pediatric Respiratory Diseases – A Comprehensive Textbook is an essential reference for the proper clinical approach to respiratory diseases in children. It is intended for all interns, residents and fellows with interest in pediatric pulmonary medicine, as well as practicing physicians, general practitioners, pediatricians and pulmonologists who face pediatric respiratory disorders in daily clinical practice. |
| cystic fibrosis enzyme replacement therapy: Cystic Fibrosis Stephanie Duggins Davis, Margaret Rosenfeld, James Chmiel, 2020-05-21 This book provides a comprehensive overview of the multisystem disease, cystic fibrosis, for both pediatric and adult patients. Written by experts in the field, the text outlines the progressive nature of CF as well as the impact of this autosomal recessive disease on the respiratory, gastrointestinal, endocrine, rheumatologic, and renal systems, as well as the patient’s mental health. The book begins with a chapter describing the history of cystic fibrosis and how the face of this life-shortening disease has changed over the past several decades. The following chapters elucidate the pathophysiology of how cystic fibrosis impacts each organ system. Current management and therapeutics are detailed with step-by-step guidelines for clinicians. This book is unique in that it highlights the entire person, not just the respiratory system, with detailed inclusion of the patient perspectives throughout, informing practice standards and considerations. This is an ideal guide for pediatric and adult physicians who care for patients with cystic fibrosis, as well as respiratory therapists, physical therapists, nurses, nutritionists, and pharmacists who care for these patients. |
| cystic fibrosis enzyme replacement therapy: Neuromuscular Disorders: Management and Treatment E-Book Tulio E. Bertorini, 2010-09-08 Neuromuscular Disorders presents a multi-disciplinary approach to the management and therapeutic treatment of the full range of neuromuscular disorders and resulting complications. Dr. Tulio Bertorini and a contributing team of the world’s leading authorities in the field provide the latest tools and strategies for minimizing disability and maximizing quality of life. Effectively treat your patients using the latest management tools and targeted therapeutic strategies. Manage all neuromuscular disorders as well as resulting complications through comprehensive coverage of diagnosis and evaluations, treatments, and outcomes. Apply the multi-disciplinary approach of an expert in clinical neuromuscular care and a team of world-renown contributors. Easily refer to tools for diagnosis, treatment algorithms, and drug tables included throughout the text. |
| cystic fibrosis enzyme replacement therapy: Chronic Lung Diseases Sheikh Rayees, Inshah Din, Gurdarshan Singh, Fayaz A Malik, 2020-07-20 Chronic lung diseases: pathophysiology and therapeutics provides a much-needed reference tool for pathologists, practicing pulmonologists and researchers who are currently working on lung related diseases. Each chapter addresses a specific lung disease, which it introduces before turning to the disease’s pathophysiology, current treatment and future prospects. Various key lung diseases are covered, including chronic obstructive pulmonary disease, lung cancer, tuberculosis, chronic pneumonia, acute respiratory distress syndrome, asthma, cystic fibrosis and pulmonary hypertension. Medical students and researchers whose work involves pulmonary related disorders will find this work both instructive and informative. |
| cystic fibrosis enzyme replacement therapy: Kendig and Chernick's Disorders of the Respiratory Tract in Children E-Book Robert W. Wilmott, Thomas F. Boat, Andrew Bush, Victor Chernick, Robin R Deterding, Felix Ratjen, 2012-02-25 Kendig, Chernick’s Disorders of the Respiratory Tract in Children is the definitive medical reference book to help you confront critical challenges using the latest knowledge and techniques. You’ll get the state-of-the-art answers you need to offer the best care to young patients. Tackle the toughest challenges and improve patient outcomes with coverage of all the common and rare respiratory problems found in newborns and children worldwide. Get a solid foundation of knowledge to better understand and treat your patients through coverage of the latest basic science and its relevance to clinical problems. Get comprehensive, authoritative coverage on today’s hot topics, such as interstitial lung disease, respiratory disorders in the newborn, congenital lung disease, swine flu, genetic testing for disease and the human genome, inflammatory cytokines in the lung, new radiologic techniques, diagnostic imaging of the respiratory tract, and pulmonary function tests. Learn from the experts with contributions from 100 world authorities in the fields of pediatrics, pulmonology, neurology, microbiology, cardiology, physiology, diagnostic imaging, anesthesiology, otolaryngology, allergy, and surgery. |
| cystic fibrosis enzyme replacement therapy: Bioactive Food as Dietary Interventions for Liver and Gastrointestinal Disease Ronald Ross Watson, Victor R Preedy, 2012-10-22 Bioactive Food as Dietary Interventions for Liver and Gastrointestinal Disease provides valuable insights for those seeking nutritional treatment options for those suffering from liver and/or related gastrointestinal disease including Crohn's, allergies, and colitis among others. Information is presented on a variety of foods including herbs, fruits, soy and olive oil. This book serves as a valuable resource for researchers in nutrition, nephrology, and gastroenterology. - Addresses the most positive results from dietary interventions using bioactive foods to impact diseases of the liver and gastrointestinal system, including reduction of inflammation, improved function, and nutritional efficiency - Presents a wide range of liver and gastrointestinal diseases and provides important information for additional research - Associated information can be used to understand other diseases, which share common etiological pathways |
| cystic fibrosis enzyme replacement therapy: Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics Reed E. Pyeritz, Bruce R. Korf, Wayne W. Grody, 2024-10-23 For decades, Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics has served as the ultimate resource for clinicians integrating genetics and genomics into medical practice. With detailed coverage in contributions from more than 250 of the world's most trusted authorities in medical genetics and a series of 11 volumes available for individual sale, the Seventh Edition of this classic reference includes the latest information on seminal topics such as prenatal diagnosis, genome sequencing, public health genetics, genetic counseling, and management and treatment strategies to complete its coverage of this growing field for students, health providers, and researchers involved in the care of patients with genetic conditions, and increasingly, all areas of health and disease. This comprehensive yet practical resource emphasizes theory and research fundamentals related to the applications of medical genetics and genomics across the full spectrum of inherited disorders and applications to medicine more broadly. In this volume, leading physicians and researchers thoroughly examine medical genetics and genomics as applied to developmental disorders, as well as genetic conditions that affect hearing and vision. Here genetic researchers, students, and health professionals will find new and fully revised chapters on human developmental genetics, disorders affecting craniofacial development, chromosomal abnormalities, including aneuploidies and structural abnormalities, hereditary hearing impairment, and various genetic conditions of the eye. With regular advances in genomic technologies propelling precision medicine into the clinic, Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics, Seventh Edition bridges the gap between high-level molecular genetics and practical application and serves as an invaluable clinical tool for health professionals and researchers. · Thoroughly introduces genetic researchers, students, and healthcare professionals to the principles of human developmental genetics · Examines a wide range of developmental disorders, including craniofacial development as well as disorders affecting hearing and vision · Includes color images supporting identification, concept illustration, and method processing · Features contributions by leading international researchers and practitioners of medical genetics |
| cystic fibrosis enzyme replacement therapy: Facts about Cystic Fibrosis , 1995 |
| cystic fibrosis enzyme replacement therapy: History of the Pancreas: Mysteries of a Hidden Organ John M. Howard, Walter Hess, 2012-12-06 Never before has a comprehensive history of the pancreas like History of the Pancreas been published. It not only is a historical review of the science of medicine, it is liberally interspersed with anecdotal vignettes of the researchers who have worked on this organ. Much of it, such as the discovery of the duct of Wirsüng, of the islets of Langerhans, of insulin, gastrin and their tumors, reads like the adverture, which it is. This book, divided into 14 chapters, is written in a narrative style and is easily readable, as glimpses of the investigators, those who failed as well as those who succeeded, adds both perspective and human interest. Each chapter is completely referenced, totaling over 1500 references. As a reference book for students, teachers, investigators, writers, its detailed hjistorical documentation is unique. From the pre-Christian era of Asia Minor, to Greece, Rome, Europe and America, to the explosive progress in Japan, the history is there. History of the Pancreas: Mysteries of a Hidden Organ fills a gap. |
| cystic fibrosis enzyme replacement therapy: The Cystic Fibrosis Transmembrane Conductance Regulator Kevin L. Kirk, David C. Dawson, 2003-10-31 The CFTR chloride channel is one of the most well studied transport proteins in biology. Yet there remain many mysteries about the functional properties and biological roles of this ABC transporter. The Cystic Fibrosis Transmembrane Conductance Regulator addresses a select series of `hot' topics that relate to the function of CFTR, and the links between CFTR dysfunction and human disease (i.e., cystic fibrosis). The timeliness of these topics distinguishes this collection from previous volumes of this type. Given the general interest in CFTR, this collection will appeal to a broad readership with interests in CFTR, cystic fibrosis, ion channels and ABC transporters. |
| cystic fibrosis enzyme replacement therapy: National Institute of Arthritis and Metabolic Diseases National Institute of Arthritis and Metabolic Diseases (U.S.), 1969 |
| cystic fibrosis enzyme replacement therapy: Cystic Fibrosis in Primary Care Douglas Lewis, MD, FAAFP, 2020-01-28 Cystic fibrosis is a disease that affects the entire body. It tends to be thought of as primarily a pulmonary disease since pulmonary decline is the main factor in early mortality. Because of the multi-system nature of the disease, a better understanding of cystic fibrosis expands the family physician's understanding of subjects ranging from genetics to pulmonary function to nutrition to colon transport to hydration to electrolyte management. The primary care philosophy is unique in that it always considers how a narrow problem can affect an individual globally. Cystic Fibrosis care can often feel fractured to patients as they are sent to multiple specialists to deal with problems outside of the comfort level of a prior or current specialist. With a broad medical philosophy, care is more inclusive as clinicians can manage topics such as diabetes and preventive care without multiple referrals and additional appointments. Family physicians are well-positioned and well-qualified to competently meet many of the care needs of those with cystic fibrosis. This book is edited by a family medicine physician who has specialist level experience with the disease. It opens with a background on cystic fibrosis foundations and centers to familiarize the reader. The next chapter gives a basic overview of the disease. Each of the subsequent chapters provide a comprehensive look at how cystic fibrosis affects other areas of the body that the primary care physician should be familiar with. Major components of cystic fibrosis such as physiology, spirometry, inflammation, airway clearance, chronic infection, cystic fibrosis related diabetes and pancreatic insufficiency, among others, are thoroughly explained. Written by experts in the field, Cystic Fibrosis in Primary Care appeals to all family physicians as well as specialists, residents, medical students physician assistants and nurse practitioners alike. |
| cystic fibrosis enzyme replacement therapy: Rare Diseases and Orphan Products Institute of Medicine, Board on Health Sciences Policy, Committee on Accelerating Rare Diseases Research and Orphan Product Development, 2011-04-03 Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development. |
| cystic fibrosis enzyme replacement therapy: In Vitro Methods in Pharmaceutical Research Jose V. Castell, Maria Jose Gmez-Lechn, 1996-10-04 In Vitro Methods in Pharmaceutical Research provides a comprehensive guide to laboratory techniques for evaluating in vitro organ toxicity using cellular models. Step-by-step practical tips on how to perform and interpret assays for drug metabolism and toxicity assessment are provided, along with a comparison of different techniques available. It is a welcome addition to the literature at a time when interest is growing in cellular in vitro models for toxicology and pharmacology studies. - Meets the continuing demand for information in this field - Compares In Vitro techniques with other methods - Describes cell-culture methods used to investigate toxicity in cells derived from different organs - Includes contributions by leading experts in the field |
Update in Enzyme Therapy for Cystic Fibrosis - Stanford …
Pancreatic Enzyme Replacement Therapy (PERT) Implemented in adults and children who are pancreatic insufficient Approximately 85-90% of patients with CF
ANG003 is a Novel Non-Porcine Enzyme Replacement for …
Jul 30, 2024 · study of ANG003, a novel broad-spectrum orally delivered non-porcine enzyme replacement therapy, in people with CF who have exocrine pancreatic insufficiency (EPI). …
Pancreatic enzyme replacement therapy (PERT) for adults with …
Who needs Pancreatic Enzyme Replacement Therapy? About 85% of adults with Cystic Fibrosis (CF) are pancreatic insufficient. This means the pancreas is unable to produce or release …
P347 Management of Pancreatic Enzyme Replacement …
replacement therapy dosages were documented from patient files. Results: The mean age of the patients in the study is 8.8 ± 4.4 years, with 42 (51.9%) of the patients is male.
Pancreatic enzyme replacement therapy in cystic fibrosis: dose ...
Objectives: Pancreatic enzyme replacement therapy (PERT) remains a backbone in the nutritional treatment of cystic fibrosis. Currently, there is a lack of an evidence-based tool that allows …
Title: A Long-Term Prospective Observational Safety Study of …
to study the incidence of FC in cystic fibrosis patients to see if there may be a link between taking pancreatic enzyme medications and developing FC. The study will be conducted for a total of …
Dosing regimens for pancreatic enzyme replacement therapy …
In CF, there is an absent or dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein caused by a gene mutation, which can lead to dysfunctional secretion in
2. SYNOPSIS: INVESTIGATIONAL PLAN - AbbVie
requirements for the use of pancreatic enzyme replacement therapy (PERT) for the treatment of exocrine pancreatic insufficiency, including a 10-year, observational study to prospectively …
Journal of Cystic Fibrosis
Cystic Fibrosis (CF) causes fat malabsorption and early on- set pancreatic insufficiency (PI) in approximately 80–90% of pa- tients [1]. Malnutrition and stunting can only be avoided by accu- …
Pancreatic Enzyme Replacement Therapy (PERT) for Children …
PERT is essential for everyone with Cystic Fibrosis (CF) with pancreatic insufficiency. This means your pancreas cannot make enough enzymes to digest food and absorb nutrients. Without …
Anagram Therapeutics Presents Positive Data from Clinical …
Sep 25, 2024 · company dedicated to improving the lives of people with cystic fibrosis (CF) and other rare diseases, today presented results from a dose-ranging study of ANG003, a novel …
Efficacy and safety of PANCREAZE® for treatment of exocrine …
Background: Pancreatic enzyme replacement therapy (PERT) is critical for correction of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). Methods: This was a …
FOR IMMEDIATE RELEASE Anagram Therapeutics Announces …
Sep 27, 2023 · exocrine pancreatic insufficiency, is a new class of broad-spectrum digestive enzyme replacement therapy in clinical trials in people with cystic fibrosis. Anagram is a …
Pancreatic enzyme replacement therapy for young cystic …
A pancreatic enzyme preparation developed particularly for infants, Creon® for children (CfC), contains smaller granules to be administered with a dosing spoon (5000 lipase units per …
Medical Nutrition Therapy for Cystic Fibrosis: Beyond …
biotics, mucus-thinning drugs, and pancreatic enzyme replacement therapy (PERT) (1). Dietary recommendations for people with cystic fibrosis include 20% to 50% more calories than …
Don’t Sweat It: Basics of CF Nutrition - Children's Hospital Los …
1. Discuss the physiology of cystic fibrosis and its nutrition related complications 2. Calculate the estimated nutrition needs and dosage of enzyme replacement therapy for children with cystic …
Timing of pancreatic enzyme replacement therapy (PERT) in …
We reviewed the evidence about the diFerent timings of pancreatic enzyme replacement therapy (PERT) in people (of any age) with cystic fibrosis (CF). Background
Study design considerations for evaluating the efficacy and …
We present in this paper recommendations of the Cystic Fibrosis Foundation’s Cystic Fibrosis (CF) Therapeutics Development Network and Data Safety Monitoring Board regarding study …
Maximal daily dose of pancreatic enzyme replacement …
The current recommendations for dosing of pancreatic enzyme replacement therapy (PERT) in infants with cystic fibrosis (CF) were made using a limited evidence base. The per meal …
Anagram Launch Release FINAL 040423
living with cystic fibrosis and other rare diseases. ANG003, Anagram’s lead product for the treatment of malabsorption and exocrine pancreatic insufficiency, is a new class of broad …
Update in Enzyme Therapy for Cystic Fibrosis - Stanford …
Pancreatic Enzyme Replacement Therapy (PERT) Implemented in adults and children who are pancreatic insufficient Approximately 85-90% of patients with CF
ANG003 is a Novel Non-Porcine Enzyme Replacement for …
Jul 30, 2024 · study of ANG003, a novel broad-spectrum orally delivered non-porcine enzyme replacement therapy, in people with CF who have exocrine pancreatic insufficiency (EPI). …
Pancreatic enzyme replacement therapy (PERT) for adults …
Who needs Pancreatic Enzyme Replacement Therapy? About 85% of adults with Cystic Fibrosis (CF) are pancreatic insufficient. This means the pancreas is unable to produce or release …
P347 Management of Pancreatic Enzyme Replacement …
replacement therapy dosages were documented from patient files. Results: The mean age of the patients in the study is 8.8 ± 4.4 years, with 42 (51.9%) of the patients is male.
Pancreatic enzyme replacement therapy in cystic fibrosis: …
Objectives: Pancreatic enzyme replacement therapy (PERT) remains a backbone in the nutritional treatment of cystic fibrosis. Currently, there is a lack of an evidence-based tool that allows …
Title: A Long-Term Prospective Observational Safety Study …
to study the incidence of FC in cystic fibrosis patients to see if there may be a link between taking pancreatic enzyme medications and developing FC. The study will be conducted for a total of …
Dosing regimens for pancreatic enzyme replacement therapy …
In CF, there is an absent or dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein caused by a gene mutation, which can lead to dysfunctional secretion in
2. SYNOPSIS: INVESTIGATIONAL PLAN - AbbVie
requirements for the use of pancreatic enzyme replacement therapy (PERT) for the treatment of exocrine pancreatic insufficiency, including a 10-year, observational study to prospectively …
Journal of Cystic Fibrosis
Cystic Fibrosis (CF) causes fat malabsorption and early on- set pancreatic insufficiency (PI) in approximately 80–90% of pa- tients [1]. Malnutrition and stunting can only be avoided by accu- …
Pancreatic Enzyme Replacement Therapy (PERT) for …
PERT is essential for everyone with Cystic Fibrosis (CF) with pancreatic insufficiency. This means your pancreas cannot make enough enzymes to digest food and absorb nutrients. Without …
Anagram Therapeutics Presents Positive Data from Clinical …
Sep 25, 2024 · company dedicated to improving the lives of people with cystic fibrosis (CF) and other rare diseases, today presented results from a dose-ranging study of ANG003, a novel …
Efficacy and safety of PANCREAZE® for treatment of …
Background: Pancreatic enzyme replacement therapy (PERT) is critical for correction of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). Methods: This was a …
FOR IMMEDIATE RELEASE Anagram Therapeutics …
Sep 27, 2023 · exocrine pancreatic insufficiency, is a new class of broad-spectrum digestive enzyme replacement therapy in clinical trials in people with cystic fibrosis. Anagram is a …
Pancreatic enzyme replacement therapy for young cystic …
A pancreatic enzyme preparation developed particularly for infants, Creon® for children (CfC), contains smaller granules to be administered with a dosing spoon (5000 lipase units per …
Medical Nutrition Therapy for Cystic Fibrosis: Beyond …
biotics, mucus-thinning drugs, and pancreatic enzyme replacement therapy (PERT) (1). Dietary recommendations for people with cystic fibrosis include 20% to 50% more calories than …
Don’t Sweat It: Basics of CF Nutrition - Children's Hospital …
1. Discuss the physiology of cystic fibrosis and its nutrition related complications 2. Calculate the estimated nutrition needs and dosage of enzyme replacement therapy for children with cystic …
Timing of pancreatic enzyme replacement therapy (PERT) in …
We reviewed the evidence about the diFerent timings of pancreatic enzyme replacement therapy (PERT) in people (of any age) with cystic fibrosis (CF). Background
Study design considerations for evaluating the efficacy and …
We present in this paper recommendations of the Cystic Fibrosis Foundation’s Cystic Fibrosis (CF) Therapeutics Development Network and Data Safety Monitoring Board regarding study …
Maximal daily dose of pancreatic enzyme replacement …
The current recommendations for dosing of pancreatic enzyme replacement therapy (PERT) in infants with cystic fibrosis (CF) were made using a limited evidence base. The per meal …
Anagram Launch Release FINAL 040423
living with cystic fibrosis and other rare diseases. ANG003, Anagram’s lead product for the treatment of malabsorption and exocrine pancreatic insufficiency, is a new class of broad …
